Introduction: Prader-Willi-Syndrom (PWS) is a rare multisystem genetic disorder characterized by hypothalamic-pituitary dysfunction. Various characteristic clinical features and several endocrinological problems such as hypogonadism, hypothyroidism, Growth hormone (GH) and adrenal deficiency have been described. Since GH-Therapy (GHT) was approved the physical benefits of the treatment have been established in many studies. It is now recommended to start treatment as soon as possible. The aim of this study is to shed light on possible differences in height, carbohydrate and lipid metabolism between children with PWS in whom GHT was initiated either during or after their first year of life. Patients and Methods: This retrospective longitudinal study included 62 children (31 males) with genetically confirmed PWS in whom fasting morning blood samples and auxological parameters were obtained before start of therapy and semi-annually thereafter. The early treatment cohort A consisted of 21 (11 males) infants who were recruited at 0.3–0.99 yrs (mean 0.72 yrs) for GHT. The later treatment cohort B entailed 41 individuals (20 males) in whom GHT was initiated at 1.02–2.54 yrs (mean 1.42 yr). Results: Auxology: Mean length/height-SDSPWS differed significantly throughout the entire observation period between the groups: 1 yr: A: 0.37 (±0.83) vs B: 0.05 (±0.56); 5 yrs: A: 0.81 (±0.67) vs. B: 0.54 (±0.64); p=0.012). No significant differences were found in BMI, lean body mass or percent body fat. Endocrinological Parameters: Mean IGF-I SDS in group A did not differ significantly from group B and mean IGF-I SDS were mostly below 0 SDS (within normal range) in both groups.Lipid Metabolism: Low-density lipoprotein (LDL) was statistically significantly lower in Group A than in Group B during the entire course of the study (LDL: 1 yr: A: 79 (±20) mg/dl vs. B: 90 (±19) mg/dl; 5 yrs: A: 91(±18) mg/dl vs. 104 (±26) mg/dl; p=0.024).Carbohydrate Metabolism: Significant differences in mean fasting insulin levels and HOMA-IR between the two groups were found (fasting insulin p=0.012; HOMA-IR p=0.006). Significant differences in HbA1c and blood glucose levels were also determined between the two groups (HbA1c: p<0.001; blood glucose: p=0.022). Conclusion:Our analysis shows that early GHT had a statistically significant favorable effect on height-SDS, LDL, HOMA-IR and fasting insulin. The two groups did not significantly differ in BMI-SDS, body composition or IGF-I SDS.
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